Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...

(槟城8日讯) 患有轻度型儿童脊髓性肌肉萎缩症（Spinal Muscular Atrophy,SMA type ...

Scholar Rock aims for a 2025 launch of apitegromab in SMA, expecting $2 billion in global revenue and significant growth from upcoming trials and favorable data.

Back to Healio Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease ...

Positive results from its Phase III STEER study mean Novartis could boost sales of its OAV101 IT drug for spinal muscular ...

Creatinine, a byproduct of muscle function, may be more sensitive in tracking SMA progression and response to treatment than ...

The phase 3 STEER study enrolled 127 treatment-naïve patients aged 2 to less than 18 years with SMA type 2 who were able to sit but had never walked independently.

Crystal Rondeau’s muscle wasting disease has left her with only one finger to press an alarm button or send a text. The ...

Without this protein, motor neurons deteriorate, leading to muscle weakness, impaired movement, and eventually respiratory ...

The Phase 3 STEER trial enrolled more than 100 children with SMA type 2, ages 2-17, who were able to sit, but hadn't walked ...